Ruijian Pharmaceutical "Qingqi" received the first IND acceptance of pluripotent stem cell-derived drug in the field of Parkinson's disease in China

On April 11, 2022, the world ushered in the 25th "World Parkinson's Disease Day".
According to the first discovery of Parkinson's disease, a common neurological disorder, it has been more than 200 years
.

At present, there are more than 3 million Parkinson's disease patients in China, of which the prevalence rate of people over 65 years old is 1.
7%
.

Faced with the vast number of Parkinson's patients, new drug R&D companies are also constantly exploring relevant solutions for this disease
.

On the "World Parkinson's Day" in 2022, Wuhan Ruijian Pharmaceutical Technology Co.
, Ltd.
(referred to as "Ruijian Pharmaceutical") officially submitted an IND application for the Parkinson's R&D pipeline NouvNeu001 to the National Center for Drug Evaluation.
The review center officially accepted the application
.

Preclinical data show that through the self-developed high-efficiency chemical small molecule-induced functional cell regeneration technology, NouvNeu001 can be efficiently transformed into mature dopaminergic neurons after transplantation into the body, secrete dopamine transmitters, and form with the original neurons in the body.
Neural connections, resulting in a comprehensive therapeutic function to improve Parkinson's symptoms
.

It provides a brand-new cell drug clinical treatment path for Parkinson's disease
.

It is worth mentioning that the acceptance of the NouvNeu001 IND also made Rejian Pharmaceuticals officially become the first biopharmaceutical company in China and the second in the world to receive clinical applications for pluripotent stem cell-derived drugs in the Parkinson's field, second only to Bayer.
owned by BlueRock
.

1 The IND acceptance of the research pipeline has brought "new hope" for Parkinson's cure.
"In addition to hand tremors, numbness, dizziness, stiffness, and loss of language control, he tried his best to cover up his symptoms, but he had to face his own problems.
The fact of entering the kingdom of disease"
.

Helmut Dubier, who was a professor of sociology at the University of Giessen, used tenacious brushstrokes to describe his real life after suffering from Parkinson's in "Hello, Parkinson"
.

All of this stems from the massive death of dopaminergic neurons in the brain that secrete dopamine transmitters
.

Unfortunately, even though the pathological characteristics of Parkinson's disease are very clear, there is still no effective clinical means to reverse the trend of continuous death of dopaminergic neurons in the brain
.

With the deepening of the aging trend of the population, the incidence of neurodegenerative diseases such as Parkinson's disease is also rapidly increasing, which not only seriously affects the quality of life of patients, but also imposes a heavy burden on family members and national medical insurance
.

The successful acceptance of the NouvNeu001 IND application will undoubtedly bring new hope for the clinical treatment of Parkinson's disease
.

As the world's first high-tech pharmaceutical company focusing on the research of high-efficiency chemical small molecules to induce functional cell regeneration, Ruijian Medicine has established an induced pluripotent stem cell (iPSC) product development platform based on regenerative medicine
.

NouvNeu001 uses this platform to use induced pluripotent stem cells as "seeds" to cultivate healthy dopamine neural precursor cells, which are then implanted into the lesion to make up for the lack of dopaminergic neurons
.

In addition to the secretion of dopamine and other transmitters, these precursor cells can further secrete a variety of proteins and small nucleic acids to improve Parkinson's disease and provide a good "soil" environment for cells transplanted to the brain to function
.

Based on the above strategies, NouvNeu001 can effectively promote the recovery of patients' movement and other functions
.

"Traditional drug and surgical treatments have great limitations for many diseases, and cell therapy's unique treatment approach is a beneficial complement to traditional treatments
.

"Dr.
Yan Xuan, the innovative investment partner of Changjiang Securities who participated in the A+ round of financing of Ruijian Pharmaceutical, commented
.

Judging from the results of the completed pharmacological efficacy test and safety test, all kinds of experimental animals using NouvNeu001 were treated with NouvNeu001 for four weeks.
, not only the behavioral function has been greatly restored, but many of its physiological and biochemical indicators have also been significantly improved
.

In contrast, similar drugs worldwide for Parkinson's disease require the shortest 12 weeks of administration to start.
The pharmacological efficacy of Ruijian NouvNeu001 has significant advantages
.

"The successful acceptance of the NouvNeu001 IND application reflects the efficient execution of the Ruijian pharmaceutical team
.

In the process of research and development, we fully consider the unmet clinical needs, always take the interests of patients as the main goal, and conduct full scientific and strict safety evaluations on the products
.

"Dr.
Wei Jun, co-founder and CEO of Ruijian Pharmaceutical, said
.

2 The potential of chemical-induced platform companies burst out.
Behind the acceptance of the IND application of Parkinson's research pipeline is the compound R&D centered on chemical induction built by Ruijian Pharmaceutical .
system, and established a number of international first technology platforms
.

At present, using the multi-platform modularization and flexible integration method, Ruijian Pharmaceutical is creating an innovative R&D model with multiple drug forms, multiple pipelines and multiple drivers centered on the core data platform , and quickly completed the layout of several R&D pipelines, including NouvNeu001, which was successfully submitted to the IND this time, with great ductility
.
According to the
company's official website, Ruijian Pharmaceutical has quickly completed a number of R&D pipelines in just over four years since its establishment.
R&D pipeline layout
.

In addition to cell-based drugs for Parkinson's disease, spinal cord injury, stroke and other neurodegenerative diseases, the company is also exploring cell-based drug therapy in the fields of optic neuropathy, diabetic retinopathy, amyotrophic lateral sclerosis, and liver metabolism.
the possibility of
.

The value of related technology platforms does not stop there.
According to Wei Jun, the technology platform built by the company also includes a multi-dimensional integrated database of "compound-protein target-tissue development-disease treatment", and uses network visualization, knowledge question and answer , machine learning models and other tools, and the construction of a metadata integration platform (iReMeta), which is of great significance for revealing the key molecules and targets of the human developmental map and clarifying the mechanism of disease treatment
.

In addition, in the face of complex drug development in the future, Ruijian Medicine has also established a high-throughput gene editing platform, iReDita, including various gene editing tools such as CRISPR and TALEN
.

In addition to the current chemically-induced cell drug platform, which is becoming more and more perfect, Regent has also built a chemically-induced complex drug development platform (iReCena), which aims to tap the therapeutic potential of cell derivatives in various indications
.

 Figure 1.
The pharmaceutical system of Ruijian Pharmaceutical relies on several international first technology platforms developed by the company.
Ruijian Pharmaceutical has become a platform company, and the advantages of the self-developed technology platform are gradually transformed into the competitive advantages of the company's development
.

According to public information, Ruijian Pharmaceutical has successively won two rounds of financing of nearly 100 million yuan in the A round and A+ rounds in less than half a year from August 2021 to January 2022
.

And before the A+ round of financing landed, signed a strategic cooperation agreement with Danaher Life Sciences China to carry out close cooperation in the field of global breakthrough cell drug development and production
.

According to the agreement, the two parties will jointly build the "Ruijian Pharmaceutical Technology Co.
, Ltd.
-Danaher China Life Science Joint Laboratory" to jointly promote the research and development and clinical application of a globally innovative AI-based chemically induced cell therapy
.

In addition, while receiving multiple rounds of financing, Ruijian Pharmaceutical also officially moved to a new R&D and production site with a total area of ​​4,000 square meters in August last year, completed the company's site and hardware upgrades, and accelerated pipeline research and development
.

The company will also continue to improve the whole-process innovative R&D and production system.
Through the cooperation of upstream and downstream industry chains and the co-construction of demonstration laboratories with Danaher, the company will create an industrial-grade integrated design and configuration benchmark for cell drugs
.

"Actually, in the process of this IND, Ruijian Pharmaceutical has simultaneously completed a complete set of clinical ready-to-use technical reserves, fully considering the product design of storage and logistics stability, and taking into account international supply and logistics requirements," Wei Jun said
.

Figure 2.
Ruijian Pharmaceutical Industrial Production Layout